November 5, 2015 9:50am
BLUE says 2 of 3 beta-thalassemia major patients with B0/B0 genotype treated with LentiGlobin. BB305 received single transfusion after discharge and third remains transfusion-dependent.
All 4 non-B0/B0 patients in the “Northstar” study have been transfusion-free for at least 90 days, median 287 days
Median HbA T87Q expression among 7 patients is 5.2 g/dL; total hemoglobin ranged from 8.5-11.1 g/dL at last visit
Data reflects patients monitored for at least 6 months post-infusion
BLUE CEO Nick Leschly said in phone interview that one-third of U.S. and European patients with beta-thalassemia major have two copies of defective geneSource: Bloomberg by Doni Bloomfield and Catherine Larkin)
The Bottom Line: In “HGB-205” study, both patients with beta-thalassemia B0/BE genotype have remained transfusion-free for at least 15 months; one additional beta-thalassemia patient had only one month follow-up post-infusion
Patient with severe sickle disease is producing ~51.5 anti-sickling hemoglobin at 9 months post-infusion, hasn’t had a post-treatment hospitalization for disease related event despite stopping chronic transfusions at day 88. <NOTE: Patient was producing 45% anti-sickling hemoglobin at 6 months, according to 6/13 release.>
BLUE closed at $90.15 and is DOWN -$16.14 or -17.90% to $74.01 in early trading (first 20 minutes)
In “HGB-206” study, 1 patient with sickle cell disease has been infused, no grade 3+ drug-related adverse events
Findings will be presented at American Society of Hematology meeting in Dec.
